AAV1-FGF21

AAV1-FGF21 (also known as KRIYA-497 or VV-14303) is an investigational, one-time intramuscular gene therapy designed to enable continuous, long-term expression of native fibroblast growth factor 21 (FGF21). Originally developed by researchers at the Universitat Autònoma de Barcelona (UAB) and spun out into Tramontane Therapeutics, the program was acquired by Kriya Therapeutics in September 2023. The therapy utilizes an adeno-associated virus serotype 1 (AAV1) vector to deliver the FGF21 coding sequence directly into skeletal muscle, turning muscle tissue into a biofactory that secretes functional, native FGF21 into the bloodstream. By activating the FGF receptor/β-Klotho signaling pathway in metabolic tissues, AAV1-FGF21 regulates glucose and lipid metabolism. In preclinical models, the therapy has demonstrated the ability to reverse metabolic dysfunction-associated steatohepatitis (MASH) and liver fibrosis, prevent hepatocellular carcinoma, counteract obesity and insulin resistance, and extend healthspan and lifespan in aged mice. It is currently in Phase 1/2 clinical development for the treatment of MASH.

Other names

AAV-FGF21AAV-FGF-21AAV-FGF 21AAV-mediated FGF21 gene therapyAAV1-mediated FGF21 gene therapyAAV-1-mediated FGF21 gene therapyAAV 1-mediated FGF21 gene therapy